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ACTIVE NOT RECRUITING Phase 1

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

NCT06559176 · View on ClinicalTrials.gov ↗

Study Summary

This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations in the NCF1 (Neutrophil Cytosolic Factor 1) gene.

Interventions

  • BIOLOGICAL PM359

Study Locations (5)

California

  • University of California Los Angeles Medical Center — Los Angeles

Maryland

  • NIH Clinical Center — Bethesda

Tennessee

  • The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers — Nashville

Quebec

  • CHU - Sainte Justine Hospital — Montreal

England

  • University College of London Hospital — London

Trial Details

FieldValue
Enrollment Target 12 participants
Start Date 2024-10-17
Est. Completion 2030-02
Phase Phase 1

Sponsor

Prime Medicine

29 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06559176

The ClinicalTrials.gov registry entry for NCT06559176 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Prime Medicine, which has 29 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 2 conditions, with Chronic Granulomatous Disease appearing as the primary indexed condition, and to 1 intervention — of which PM359 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT06559176 reports 5 study locations spanning 5 distinct geographic areas — top geographies include California, Maryland, Tennessee. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT06559176 about?

NCT06559176 is a clinical study titled "A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )". This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations i...

What is the current status of trial NCT06559176?

This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2024-10-17. Estimated completion is 2030-02.

What conditions does trial NCT06559176 study?

This clinical trial studies the following conditions: Chronic Granulomatous Disease, Granulomatous Disease, Chronic. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT06559176?

The interventions under investigation include: PM359 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT06559176?

This trial is sponsored by Prime Medicine, which has 29 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT06559176 being conducted?

This trial has 5 study locations across California, Maryland, Tennessee, Quebec, England. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial